Summary of ZVRA Q4 2025 Earnings Call

1Key Financial Results and Metrics

Q4 2025 Revenue: $34.1 million, with $26.4 million from MIPLYFFA sales.

Full Year 2025 Revenue: $106.5 million, primarily driven by MIPLYFFA ($87.4 million).

Net Income: Q4 net income was $12.2 million ($0.20 per share), a significant improvement from a net loss of $35.7 million in Q4 2024. Full year net income was $83.2 million ($1.40 per share), compared to a net loss of $105.5 million in 2024.

Operating Expenses: Q4 operating expenses were $23 million, down $1.5 million year-over-year. R&D expenses decreased to $2.6 million, while SG&A expenses increased to $20.4 million due to launch-related investments.

Cash Position: As of December 31, 2025, cash and equivalents totaled $238.9 million, with total debt at approximately $61.9 million.

2Strategic Updates and Business Highlights

MIPLYFFA Performance: Strong commercial performance with 52 prescription enrollment forms in 2025, including 24 in Q4. Total enrollments since launch reached 161.

Market Expansion: The company is pursuing a patent term extension for MIPLYFFA and expanding access through a global expanded access program (EAP) with 113 patients enrolled.

European Market: Submitted a marketing authorization application to the European Medicines Agency (EMA) and is prepared to respond to regulatory questions. The company is also building relationships with key opinion leaders in Europe.

Pipeline Development: Advancing Celiprolol for Vascular Ehlers-Danlos Syndrome (VEDS) with 52 patients enrolled in the ongoing Phase III DISCOVER study.

3Forward Guidance and Outlook

Growth Drivers: Anticipated continued growth in MIPLYFFA enrollments, particularly from newly diagnosed patients. The company expects variability in revenue due to the nature of the ultra-rare disease market.

Market Potential: Management is optimistic about the addressable market for NPC, estimating between 300-900 diagnosed patients in the U.S. and continuing efforts to identify newly diagnosed patients.

Investment Focus: Plans to prioritize investments in high-impact initiatives while exploring opportunities for pipeline expansion.

4Bad News, Challenges, or Points of Concern

Enrollment Variability: The company acknowledged potential variability in patient enrollment and revenue due to the nature of the ultra-rare disease market.

Competition: Awareness of potential competition from other therapies, such as Mandos' adrabetadex for infantile onset NPC, which could impact MIPLYFFA's market position.

Regulatory Risks: While the company is prepared for EMA questions, any unforeseen regulatory hurdles could delay market access in Europe.

5Notable Q&A Insights

Patient Identification: Management highlighted ongoing efforts to identify newly diagnosed patients through disease awareness campaigns and collaborations with genetic testing companies.

Persistency Data: While it is too early to provide specific data on patient persistency on MIPLYFFA, refill rates are encouraging, indicating strong adherence.

EAP Revenue Clarification: Q4 EAP revenue was driven by named patient sales, with expectations for variability in future quarters as the program stabilizes.

Market Dynamics: The company is confident in its ability to capture a significant share of the diagnosed NPC patient population, with estimates suggesting they have engaged 40-50% of diagnosed patients. Overall, Zevra's Q4 2025 results reflect strong financial performance and strategic initiatives aimed at expanding access to MIPLYFFA while navigating challenges inherent in the rare disease market.